(166) Iterative High-throughput Screening Identifies Nanoparticles with Improved Delivery Efficiency

Introduction: Neurofibromatosis Type 1 (NF1) is caused by the loss of function of the neurofibromin protein, a tumor suppressor and down-regulator of the RAS signaling pathway. Gene replacement to reconstitute functional neurofibromin presents a promising treatment modality which, due to its wide applicability, has high potential for near-term clinical translation. However, viral vectors do not have the capacity to encapsulate the large NF1 plasmid. To overcome these challenges, we used cationic diblock polymeric nanoparticles (PNPs) to encapsulate and deliver the full length NF1 gene.